Speak Foundation Introduces Patient-Led National LGMD Centers of Excellence to Drive Clinical Trials and Innovative Treatments
Speak Foundation has announced the launch of a major new initiative aimed at transforming care delivery and accelerating therapeutic development for patients living with limb-girdle muscular dystrophy. The newly established LGMD Centers of Excellence network brings together leading academic medical institutions across the United States to create a coordinated, multidisciplinary framework for patient care while strengthening the infrastructure needed to support clinical research and emerging therapies.
Limb-girdle muscular dystrophy is not a single disease but a group of more than 30 genetically distinct disorders characterized by progressive muscle weakness, particularly affecting the hips and shoulders. These conditions vary widely in severity, age of onset, and genetic cause, making diagnosis and management especially complex. Historically, patients with LGMD have faced significant challenges, including delayed diagnosis, limited access to specialized care, and a lack of effective treatment options. As a result, care has often been fragmented across multiple providers, with limited coordination between clinical specialties.
However, the landscape for LGMD is beginning to change. Advances in molecular biology and genetic research have led to the development of targeted therapies, including small molecules, pathway-modifying drugs, and gene therapies. Several of these candidates are now progressing through clinical development, raising the possibility that disease-modifying treatments could soon become available for certain LGMD subtypes. This progress has brought renewed urgency to the need for a robust and coordinated clinical infrastructure capable of supporting both patient care and clinical trials.
The LGMD Centers of Excellence initiative is designed to address this gap. By creating a national network of specialized centers, the program aims to standardize care, improve patient access to expertise, and ensure readiness for participation in clinical trials. Each center within the network will integrate multidisciplinary teams that may include neurologists, geneticists, cardiologists, pulmonologists, physical therapists, and other specialists. This collaborative model is intended to provide comprehensive, patient-centered care that evolves alongside scientific and therapeutic advancements.
A key feature of the initiative is the inclusion of dedicated LGMD Care Coordinators at participating institutions. These professionals will play a central role in helping patients navigate the healthcare system, coordinating appointments across specialties, facilitating genetic testing where appropriate, and ensuring continuity of care over time. For patients and families who often face complex and fragmented care pathways, this level of coordination represents a meaningful improvement in the overall care experience.
According to Kat Bryant Knudson, founder of the Speak Foundation and a person living with LGMD, the initiative is rooted in both personal experience and a deep understanding of the systemic challenges facing the LGMD community. She noted that historically, therapeutic development in LGMD has been hindered not only by small patient populations but also by the lack of coordinated clinical infrastructure. By building a network of centers with standardized practices and shared goals, the initiative seeks to prepare the field for the next phase of drug development while ensuring that patients receive the care they need.
Clinical leaders involved in the program have emphasized the importance of a coordinated approach. Physicians working in neuromuscular care frequently encounter the burdens placed on families when care is fragmented across multiple providers and locations. The Centers of Excellence model addresses this issue by prioritizing integrated care, comprehensive diagnostic evaluation, and long-term follow-up. This approach is particularly important in a disease like LGMD, where clinical management must adapt over time as symptoms progress and new treatments become available.
The inaugural network includes several prominent academic medical institutions with established expertise in neuromuscular disorders. Among them is University of Iowa Health Care, where Dr. Katherine Mathews leads efforts in neuromuscular research and clinical care. VCU Health is also part of the network, home to a nationally recognized LGMD program led by Dr. Nicholas Johnson, who also founded the GRASP-LGMD initiative focused on advancing research and patient engagement.
Additional participating centers include the University of Florida College of Medicine, where Dr. Barry Byrne has been a pioneer in gene therapy approaches for muscular dystrophy, and the University of Minnesota, where Dr. Peter Kang leads the Greg Marzolf Jr. Muscular Dystrophy Center. The network also includes University of Texas at San Antonio, where Dr. Matthew Wicklund oversees clinical and research programs focused on neuromuscular diseases. Together, these institutions represent a diverse and highly experienced group of leaders in LGMD care and research.
The network is expected to expand over time to include additional centers with demonstrated expertise in LGMD. This planned growth reflects the initiative’s long-term vision of building a comprehensive national—and potentially international—framework for care and research collaboration. By connecting leading institutions, the program aims to facilitate data sharing, standardize clinical protocols, and create a unified platform for conducting multicenter clinical trials.
The launch of the LGMD Centers of Excellence comes at a pivotal moment for the field. Several therapeutic candidates targeting specific LGMD subtypes are advancing into late-stage clinical trials. Among these are treatments for FKRP-related LGMD, which may soon become the first approved therapy for a form of the disease. The potential arrival of such therapies underscores the importance of having a clinical infrastructure that can support rapid and equitable patient access.
Beyond improving care delivery, the initiative is also expected to play a critical role in accelerating drug development. Clinical trials for rare diseases often face challenges related to patient recruitment, data consistency, and long-term follow-up. By establishing a network of well-coordinated centers with standardized practices, the LGMD Centers of Excellence can help address these challenges, making it easier for researchers and sponsors to conduct high-quality studies.
The Speak Foundation has emphasized that strong clinical infrastructure is essential for translating scientific advances into real-world patient benefits. Over the past 17 years, the organization has been actively involved in building the broader ecosystem supporting LGMD research. Its efforts have included organizing scientific workshops with the U.S. Food and Drug Administration, launching the International LGMD Conference, and establishing advocacy initiatives such as LGMD Day on the Hill to engage policymakers. The foundation has also created LGMD News Magazine to amplify patient voices and highlight scientific progress.
The LGMD Centers of Excellence initiative represents the next phase of this long-term strategy. It builds on years of advocacy and collaboration by focusing on the clinical infrastructure needed to support a rapidly evolving therapeutic landscape. The program also includes partnerships with patient advocacy organizations such as CureLGMD2i and LGMD Awareness Foundation, which are contributing to efforts to expand awareness, improve patient engagement, and support community needs.
Dr. Nicholas Johnson of VCU Health highlighted the significance of the initiative for patients and families, noting that it represents a meaningful step forward in delivering coordinated care while enhancing readiness for clinical trials and access to emerging therapies. By aligning clinical excellence with patient-centered infrastructure, the program aims to better meet the needs of individuals living with LGMD.
Looking ahead, the success of the LGMD Centers of Excellence will depend on continued collaboration among patients, clinicians, researchers, and advocacy organizations. As new therapies move closer to approval, the ability to deliver them effectively will be just as important as the scientific breakthroughs themselves. Ensuring that patients can access specialized care, participate in clinical trials, and benefit from advances in treatment will require a coordinated and sustained effort.
In summary, the launch of the LGMD Centers of Excellence marks a significant milestone in the evolution of care and research for limb-girdle muscular dystrophy. By addressing longstanding gaps in clinical infrastructure and fostering collaboration across leading institutions, the initiative is poised to play a central role in improving patient outcomes and accelerating the development of new therapies. As the field continues to advance, this network provides a foundation for a more integrated, patient-centered approach to managing and ultimately treating this complex group of diseases.
About The Speak Foundation
Founded in 2008, The Speak Foundation is the first patient-led nonprofit dedicated to amplifying the voices of individuals living with all forms of Limb-Girdle Muscular Dystrophy (LGMD), accelerating research, and improving access to coordinated care. The foundation works across the research, clinical, and policy landscape to foster collaboration between patients, scientists, clinicians, and industry partners while advocating for stronger research infrastructure and therapeutic development. For more information on the LGMD Centers of Excellence, please visit thespeakfoundation.com.
About Virginia Commonwealth University and VCU Health
Virginia Commonwealth University is a major, urban public research university with national and international rankings in sponsored research. Located in downtown Richmond, VCU enrolls more than 29,000 students in more than 200 degree and certificate programs in the arts, sciences and humanities across VCU’s 12 schools and three colleges.
The VCU Health brand represents the VCU health sciences academic programs, the VCU Massey Comprehensive Cancer Center and the VCU Health System, which comprises VCU Medical Center (the only academic medical center in the region), Community Memorial Hospital, Tappahannock Hospital, Children’s Hospital of Richmond at VCU, and MCV Physicians. The clinical enterprise includes a collaboration with Sheltering Arms Institute for physical rehabilitation services. For more, please visit vcu.edu and vcuhealth.org.
About the University of Minnesota Medical School
The University of Minnesota Medical School is at the forefront of learning and discovery, transforming medical care and educating the next generation of physicians. Our graduates and faculty produce high-impact biomedical research and advance the practice of medicine. We acknowledge that the U of M Medical School is located on traditional, ancestral and contemporary lands of the Dakota and the Ojibwe, and scores of other Indigenous people, and we affirm our commitment to tribal communities and their sovereignty as we seek to improve and strengthen our relations with tribal nations. Learn more at med.umn.edu.
About University of Iowa Health Care
University of Iowa Health Care is a leading academic medical center with a nationally recognized neuromuscular program, The LGMD program is led by Dr. Katherine Mathews, joined by neuromuscular specialists Dimah Saade, Katie Lutz, Christopher Nance, Andrea Swenson and Michael Shy. The center provides comprehensive multidisciplinary care for individuals living with muscular dystrophies and other neuromuscular disorders.
Investigators at UI Health Care are actively involved in clinical research and therapeutic development aimed at advancing new treatments for LGMD. As part of the LGMD Centers of Excellence network, the team is committed to improving coordinated care and strengthening the clinical infrastructure needed to accelerate therapies.
About The University of Texas at San Antonio
The University of Texas at San Antonio is the 3rd largest university in Texas and in 2024 was rated the 6th fastest growing research university in biological sciences. The muscle disease team at UT San Antonio seeks to provide multidisciplinary care, clinical and translational research, and patient engagement for people living with muscular dystrophy. With goals of excellence in focused, personalized, and coordinated care along with groundbreaking research, our center serves patients arriving from Texas, the United States, and internationally. As one of the LGMD Centers of Excellence, we yearn to break down barriers, propel science, validate methodologies, comprehensively care for patients, and catapult treatments into the marketplace.
About University of Florida
UF Health Shands Hospital is a private, not-for-profit academic health center located in Gainesville, Florida. It serves as the primary teaching hospital for the University of Florida College of Medicine and is part of the broader UF Health system, which is one of the Southeast’s most comprehensive academic health centers.
The University of Florida pairs world class medical expertise with cutting edge advancements in gene therapies as home to the Powell Gene Therapy Center (PGTC). Led by Dr. Barry Byrne, the PGTC is working to bring innovation to care in LGMD, as well as many other inherited diseases. The team has been instrumental in administering gene therapies for LGMD and in the development of next generation therapies. At the University of Florida, patients and their families are an integral part of the process and can trust that the PGTC is relentless in pursuing the best possible outcomes for their patients – even ones yet to be imagined.
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